Okyo Pharma Limited · NASDAQ:OKYO
Okyo Pharma — Investor Presentation (July 2024)
36 slides from Okyo Pharma's investor presentation, July 2024
Slide 1: OK-101 Targeting Dry Eye Disease & Neuropathic Corneal Pain
OK -101 Targeting Dry Eye Disease & Neuropathic Corneal Pain Corporate Presentation JULY 2024 Nasdaq: OKYO
Slide 2: Disclaimer
Disclaimer By accepting and using this presentation, you will be deemed to agree not to disclose any information contained herein except as may be required by law. Additionally, certain information contained in this presentation has been obtained from published sources prepared by other parties, whi
Slide 3: Pipeline Focus: OK-101 to Treat Ocular Diseases
Pipeline Focus: OK-101 to Treat Ocular Diseases Indication Pre-clinical Phase 1 Phase 2 Phase 3 OK-101 Dry Eye Disease (“DED”) Phase 2 Trial Completed Neuropathic Corneal Pain (“NCP”) Plan to open trial in Q3 2024 Allergic Conjunctivitis Uveitis 3
Slide 4: OK-101: A Lipid-Conjugated Chemerin Peptide
OK-101: A Lipid-Conjugated Chemerin Peptide Chemerin Peptide Chemerin Receptor Linker Drug candidate with anti-inflammatory and ocular pain reducing properties Modulates Anchored Lipid Lipid conjugated peptide chemistry minimizes Inflammation drug washout and enhances the potency OK-101 pain Preserv
Slide 5: What is Dry Eye Disease?
What is Dry Eye Disease? Dry eye is a multifactorial disease of the ocular surface characterized by a loss of homeostasis of the tear film, and accompanied by ocular symptoms, in which tear film instability and hyperosmolarity, ocular surface inflammation and damage, and neurosensory abnormalities p
Slide 6: US Regulatory Requirements for Dry Eye Disease
US Regulatory Requirements for Dry Eye Disease* Dry Eye: Developing Drugs for Treatment Guidance for Industry Requirements Acceptable Signs and Symptoms of Dry Eye Sign and Symptoms (co-primaries, p<0.05, Signs: corneal staining, conjunctival staining, no unit difference required) – can be achieved
Slide 7: Current Standard of Care for DED
Current Standard of Care for DED FDA-Approved Drugs, Issues Include Slow Onset of Action, Numerous Side Effects & High Discontinuation Rate Company Labelled Onset of Primary Symptom Drug Name Primary Sign Endpoint Most common Adverse Effects (Year approved) indication Action Endpoint Restasis; Aller
Slide 8: DED Remains Unmet Need Despite Available Therapies
DED Remains Unmet Need Despite Available Therapies 90% 40% 71% 64% of 2022 prescriptions for of these prescriptions are of patients on Restasis of patients on Xiidra DED were Xiidra and new prescriptions, not discontinue treatment discontinue treatment Restasis* refills* within 3 months** within the
Slide 9: Chemerin Derived Peptide: A Potential Regulator of Inflammation & Pain
Chemerin Derived Peptide: A Potential Regulator of Inflammation & Pain Pro-inflammatory chemerin OK-101 1 activates immune cells at the 1 Stable, Protease Resistant inflammation site Chemerin Peptide Fragment Chemerin 3 Linker CHEMR23 Peptides derived from chemerin 2 Anchored Lipid 2 physiologically
Slide 10: OK-101: Targeting Chemerin Receptor
OK-101: Targeting Chemerin Receptor Chemerin Peptide Chemerin Receptor Linker Anchored Lipid Immune Cells Anti-inflammatory Response 10
Slide 11: Membrane Anchoring Improves Potency, Durability
Membrane Anchoring Improves Potency, Durability *In-vitro studies Enhanced Potency Increased Durability Human Chemerin Receptor Human Chemerin Receptor (Wash Resistant) 125 125 (Max soluble unwashed =100%) (Max soluble unwashed =100%) 100 Luciferase activity 100 Luciferase activity 75 OK-101 Anchore
Slide 12: Validation: OK-101 Efficacy in Dry Eye Mouse Model
Validation: OK-101 Efficacy in Dry Eye Mouse Model 1600 OK-101 and cyclosporine were 1400 administered topically twice a day 1200 Corneal permeability significantly Corneal Permeability 1000 reduced with OK-101 vs phosphate buffer alone 800 Potency of OK-101 was 600 comparable to cyclosporine, 400 a
Slide 13: OK-101 Normalized Goblet Cells & Reduced Inflammatory CD4 T Cells
OK-101 Normalized Goblet Cells & Reduced Inflammatory CD4 T Cells Increased Mucin-secreting Goblet Cells* Reduced Inflammatory Biomarkers* OK-101: (0.04%) normalized goblet cell density OK-101: (0.04%) reduced count of CD4+ T cells, (OK-101 was administered topically twice a day) which are known bio
Slide 14: Neuropathic Corneal Pain (NCP) in Dry Eye Disease
Neuropathic Corneal Pain (NCP) in Dry Eye Disease DED patients suffer from neuropathic corneal pain, making their condition more resistant to anti-inflammatory drugs No Current FDA approved topical treatment for neuropathic corneal pain ChemR23 receptor on leukocytes targeted by OK-101 is also expre
Slide 15: OK-101 Reduced Neuropathic Corneal Pain in Mouse Model
OK-101 Reduced Neuropathic Corneal Pain (“NCP”) in Mouse Model* Ciliary Ligation Model* Illustrates OK-101 Potential to OK-101** Reduced Corneal Pain Response Reduce Ocular Pain. Ciliary nerve ligation surgery to Comparable to Gabapentin*** (GBP) create the corneal neuropathic pain (CNP) model 70 Ve
Slide 16: OK-101 Believed to Disrupt Dry Eye Cycle
OK-101 Believed to Disrupt Dry Eye Cycle by Targeting Inflammation, Improving Tear Film Stability and by Ameliorating Multiple Symptoms OK-101 Reduces Inflammatory Markers OK-101 reduces pain, burning /stinging, blurred vision, & eye dryness OK-101 Normalizes Goblet Cell Loss Dry Eye Cycle adapted f
Slide 17: OK-101 DED Development Timeline
OK-101 DED Development Timeline Phase 2 • 240 Subjects; 80 per arm • 12 Week Dosing • 6-9 Months Duration • Potential Registration Trial 2022 2023 2024 2025 Trial Start Announced Top-Line Data CRO Partner on May 2, 2023 Announced Jan 2024 Completed Full Enrollment of 240 Patients 17
Slide 18: OK-101 DED Phase 2 Trial Design
OK-101 DED Phase 2 Trial Design ENDPOINTS Note: First Human Trial of OK-101 Primary Endpoints (through Day 85) Day -14 Day 1 Day 15 Day 29 Day 57 Day 85 Inferior Corneal Staining (Sign) Placebo run-in Ocular Discomfort Score period (Symptom) Screening Visit 1 Visit 2 Visit 3 Visit 4 Visit 5 Visit 6
Slide 19: OK-101 Phase 2 DED Study: Patient Demographics
OK-101 Phase 2 DED Study: Patient Demographics Category OK-101 (0.1%) OK-101 (0.05%) Placebo All Subjects Age (Years): n 80 81 79 240 Mean (SD) 66.2 (10.6) 63.6 (11.98) 64.4 (13.69) 64.7 (12.15) Median 67.0 63.0 65.0 65.0 Min, Max 35, 85 20, 87 26, 94 20, 94 Sex Male: n (%) 30 (37.5) 20 (24.7) 17 (2
Slide 20: Baseline Signs & Symptom of Enrolled Subjects were Balanced
Baseline Signs & Symptom of Enrolled Subjects were Balanced Sign & Symptom Baseline OK-101 (0.1%) OK-101 (0.05%) Placebo All Subjects Inferior Fluorescein Staining (0 to 4: Higher is worse) Mean (SD) 1.88 (0.554) 1.9 (0.572) 1.92 (0.574) 1.9 (0.565) Median 2.0 2.0 2.0 2.0 Min, Max 0.0, 3.0 0.0, 3.0
Slide 21: OK-101 Phase 2 DED Study: Ocular Adverse Events
OK-101 Phase 2 DED Study: Ocular Adverse Events Category OK-101 (0.1%) (N = 80) OK-101 (0.05%) (N = 81) Placebo (N = 79) Number of Ocular AEs 7 19 7 Number of Ocular TEAEs 6 16 6 Number of Ocular SAEs 0 0 0 Number of Ocular TE-SAEs 0 0 0 Number of Subjects Withdrawn Study Drug due to Ocular TEAE: n
Slide 22: Improvement in Conjunctival Sum Lissamine Green Staining
Improvement in Conjunctival Sum Lissamine Green Staining* (Sign) Data shown is change from baseline using 0-8 scale in Intent-to-Treat and Above Median Population P values are vs placebo based on ANCOVA Model Day 15 Day 29 Day 57 Day 85 Day 0 Day 15 Day 29 Day 57 Day 85 0 0 -0.2 0.05% OK-101 -0.2 LS
Slide 23: Percentage of Responders: Conjunctival Sum Lissamine Green Staining
Percentage of Responders With Improvement in Conjunctival Sum Lissamine Green Staining*, Intent-to-Treat and Above Median Population Conj Sum at least 12.5% change from Conj Sum at least 25% change from baseline in ITT baseline above median 80 60 70 52 54 50 51 51 14 14 Percentage of Responders 47 P
Slide 24: Significant Improvement in Tear Film Break Up Time
Significant Improvement in Tear Film Break Up Time1 (Sign) Data shown is change from baseline using 0-10 scale in Intent-To-Treat Population P values are vs placebo based on Wilcoxon rank sum test Day 15 Day 29 Day 57 Day 85 At least 0.5 sec change from baseline using 0-10 0.7 scale 40 0.6 30 28 LSM
Slide 25: Significant Improvement in Pain
Significant Improvement in Pain1 (Symptom) Data shown is change from baseline using 0-100 Visual Analog Scale in Intent-To-Treat Population P values are vs placebo based on Wilcoxon rank sum test Day 15 Day 29 Day 57 Day 85 VAS-Pain at least 30 unit change from baseline -6 using 0-100 Scale 35 LSM (
Slide 26: Significant Improvement in Burning/Stinging & Blurred Vision
Significant Improvement in Burning/Stinging & Blurred Vision (Symptoms1) Data shown is change from baseline using 0-100 Visual Analog Scale in Intent-To-Treat Population P values are vs placebo based on Wilcoxon rank sum test Burning/Stinging Blurred vision Day 15 Day 29 Day 57 Day 85 Day 15 Day 29
Slide 27: Responders With Improvement in Burning/Stinging and Blurred Vision
Responders With Improvement in Burning/Stinging1 and Blurred Vision1, Intent-to-Treat Population VAS-Burning Stinging at least 30 units change from VAS-Blurred Vision at least 10 unit change from baseline using 0-100 scale baseline using 0-100 scale 35 25 45 34 33 23 24 40 30 29 42% 40.7% 30.9% Perc
Slide 28: Significant Improvement in Symptoms by Patient Reported Daily Diary
Significant Improvement in Symptoms by Patient Reported Daily Diary Within First Two Weeks Pain Burning/Stinging Day 1 to 15 Day 16 to 29 Day 30 to 57 Day 58 to 85 Day 1 to 15 Day 16 to 29 Day 30 to 57 Day 58 to 85 BASELINE MEDIAN BASELINE MEDIAN CHANGE FROM CHANGE FROM Average Average Average Avera
Slide 29: Responders With Improvement in Both Signs and Symptoms at Day 85
Responders With Improvement in Both Signs*1 and Symptoms2 at Day 85 ITT Population At least 12.5% improvement in Sign and 30% improvement in Symptom change from baseline 21.9% 16 Conj Sum & BlUR VIS 26% 20 20.3% 15 Conj Sum & BURN/STING 32.9% 26 20.3% 14 Conj Sum & Pain 34.2% 26 0 5 10 15 20 25 30 3
Slide 30: OK-101 to Treat Neuropathic Corneal Pain (NCP)
OK-101 to Treat Neuropathic Corneal Pain (NCP)
Slide 31: Plans for OK-101 to Treat Neuropathic Corneal Pain (NCP)
Plans for OK-101 to Treat Neuropathic Corneal Pain (NCP) Agreement with Tufts Medical Center for NCP Phase 2 Trial Announced on July 2023 NCP Trial to be Conducted by Pedram Hamrah, MD, Professor, at Tufts Medical Center IND Cleared by FDA for OK-101 to Treat NCP Announced on Feb. 9, 2024 Phase 2 Ra
Slide 32: Plan to Start Phase 2 Trial for OK-101 to Treat NCP
Plan to Start Phase 2 Trial for OK-101 to Treat Neuropathic Corneal Pain (NCP) Phase 2, Randomized, Double masked, Placebo-Controlled Study Assessing Safety and Efficacy of OK-101 in Subjects with NCP • 48 subjects to be enrolled • Three arms, 0.05% OK-101, 0.1% OK-101 and Placebo, (16 per arms) • F
Slide 33: Patent Protection Through at Least 2039
Patent Protection Through at Least 2039 OK-101 Technology: Composition of Matter: Method of Use (Dry Eye): Use (Neuropathic Pain): US 10,233,219 US 11,197,906 US 11,254,720 Issued in US to 2034 with Issued in US to 2037 with Issued in US to 2034 (+187 potential patent term potential patent term days
Slide 34: Experienced Team With Considerable Drug Development Expertise
Experienced Team With Considerable Drug Development Expertise Management Board Gary S. Jacob, PhD, Chief Executive Officer and Director Co-inventor and Gabriele Cerrone Chairman, Founder Extensive experience founding, developer of Synergy’s FDA-approved drug Trulance, currently marketed by Bausch fi
Slide 35: Key Takeaways
Key Takeaways Significant drug effects were observed in multiple signs (conjunctival staining, TFBUT) and symptoms (ocular pain, burning/stinging and blurred vision) of DED as early as the 15-days after dosing. Drug effect was durable throughout the trial for a number of endpoints. Significant impro
Slide 36: Overcoming Limitations of Current Dry Eye Therapies
Overcoming Limitations of Current Dry Eye Therapies Thank you 36